Despite the pandemic, there were many strides in the field of medicine and research in 2020. For example, researchers first discovered and identified a rare disease called VEXAS syndrome. The…
Marian McGlockin was eighteen months old when she was diagnosed with Niemann-Pick Type C disease, a rare and fatal disorder. Marian could barely speak and was unable to walk. According…
Charcot-Marie Tooth disease (CMT) may be the most common inherited disorder of the peripheral nervous system, but that only puts the official patient count in Denmark at about 22.5 cases…
Recently, the Endocrine Society's virtual Annual Meeting, dubbed ENDO 2021, took place from March 20 - 23, 2021. During the meeting, attendees discussed the latest in endocrinology research, treatment,…
At the Endocrine Society's Annual Meeting, or ENDO 2021, Amolyt Pharma ("Amolyt") shared positive clinical data from a Phase 1 clinical trial evaluating AZP-3601 for patients with hypoparathyroidism. Although…
According to a story from Medical Xpress, an uncommon fungal infection has been discovered in Alberta, Canada. This infection, called histoplasmosis, is transmitted through the fecal matter of bats and…
In a press release from early March 2021, cell therapy company CiRC Biosciences, Inc. ("CiRC") shared that its chemically induced photoreceptor-like cells (CiPCs) received Orphan Drug designation from the FDA.…
A recent study published in Arthritis Care & Research illustrated how higher dosages of prednisone lead to better outcomes for lupus nephritis (LN) patients. After one year, participants were found to…
In a study published on pubmed.gov, an investigation of 100 patients living with progressive supranuclear palsy, a rare disorder, has helped to confirm the usefulness of a diagnostic approach called…
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